OP0252 LONG-TERM SAFETY AND EFFECTIVENESS OF CANAKINUMAB IN CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES (CAPS) – 4-YEARS DATA FROM THE RELIANCE REGISTRY
Authors: J. B. Kuemmerle-Deschner, B. Kortus-Goetze, C. Schuetz, P. Oommen, A. Janda, J. Rech, F. Weller-Heinemann, T. Kallinich, A. Pankow, G. Horneff, I. Foeldvari, P. Wasiliew, M. Borte, M. Fiene, F. Meier, T. Krickau, I. Andreica, J. Weber-Arden, N. Blank
Journal: Annals of the Rheumatic Diseases (2023)

<h3>Background</h3> CAPS (Cryopyrin-associated periodic fever syndromes) are monogenic autoinflammatory diseases causing great burden to patients due to severe systemic and organ inflammation caused by increased production of interleukin-1β (IL-1β). In clinical trials as well as in real-life, the monoclonal antibody canakinumab (CAN) effectively inhibits IL-1β and results in rapid suppression of CAPS symptoms. <h3>Objectives</h3> The RELIANCE registry explores long-term safety and effectiveness of CAN under routine clinical practice conditions in pediatric (≥2 years) and adult patients with CAPS, including Muckle-Wells syndrome (MWS), familial cold autoinflammatory syndrome (FCAS) and neonatal onset multisystem inflammatory disease (NOMID)/chronic infantile neurological cutaneous and articular syndrome (CINCA). <h3>Methods</h3> This prospective, non-interventional, observational study enrolls patients with clinically confirmed diagnoses of CAPS routinely receiving CAN. Clinical data, physician assessments and patient-reported outcomes are evaluated in 6-monthly intervals. <h3>Results</h3> 107 CAPS patients (53% female; 16% NOMID/CINCA subtypes) were enrolled by December 2022. At baseline, median age was 23 years and median duration of prior CAN treatment was 6 years. 92% of patients received greater than standard dose CAN. 75% of the patients were in disease remission at month 48 by physician assessment. Patients report stable low level disease activity (median 2.0 at baseline and 1.5 at month 48) and fatigue (median 3.0 at baseline and 1.0 at month 48). No relevant differences regarding subtype severity were observed (<b>Table 1</b>). At baseline vs. month 48, 46% vs. 50% of patients experienced impairment of social life and 32% vs. 25% reported days off from school or work due to the disease. Severe adverse events (SAE) suspected to be drug-related were reported for N=9 (8.4%) CAPS patients. The incidence rate (IR) per 100 patient-years kept stable over time. <h3>Conclusion</h3> The 48-month interim analysis of the RELIANCE registry demonstrates that long-term CAN treatment is safe and effective in patients with CAPS, independent of subtype severity. <h3>REFERENCES:</h3> NIL. <h3>Acknowledgements:</h3> NIL. <h3>Disclosure of Interests</h3> J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi, Birgit Kortus-Goetze Consultant of: Novartis, Catharina Schuetz Grant/research support from: Novartis, Prasad Oommen Grant/research support from: Novartis, Ales Janda: None declared, Jürgen Rech Speakers bureau: AbbVie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Consultant of: AbbVie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD, Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Grant/research support from: Novartis, Sobi, Frank Weller-Heinemann: None declared, Tilmann Kallinich Speakers bureau: Roche, Anne Pankow: None declared, Gerd Horneff Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp &amp; Dohme, Novartis, Pfizer, Roche, Grant/research support from: AbbVie, Chugai, Merck Sharp &amp; Dohme, Novartis, Pfizer, Roche, Ivan Foeldvari Consultant of: Novartis, Peter Wasiliew Grant/research support from: Novartis, Michael Borte Grant/research support from: Pfizer, Shire, Michael Fiene: None declared, Florian Meier Speakers bureau: Novartis, Tobias Krickau Speakers bureau: Novartis, Consultant of: Novartis, Grant/research support from: Novartis, Ioana Andreica Speakers bureau: Abbvie, Chugai, Novartis, UCB, MSD, Lilly, Sobi, Astrazeneca, Amgen, Pfizer, Gilead, Paid instructor for: Astrazeneca, UCB; Consultant Abbvie, Chugai, Novartis, UCB, Galapagos, Takeda, Astrazeneca, Lilly, Boehringer Ingelheim, Amgen, Sobi, Julia Weber-Arden Employee of: Novartis, Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi.